Special needs

Early research shows promise for new autism drug

Researchers are hopeful about a medication under development to treat symptoms of autism spectrum disorder (ASD).

Early research shows promise for new autism drug

Photo: iStockphoto

A new drug may become available for people with autism to help stimulate social and communication skills, and to mitigate repetitive behaviours and restricted interests. It’s likely several years down the road in North America, but if it comes to market, balovaptan will be the first medication targeting these ASD symptoms.

The U.S. Federal Drug Administration (FDA) just designated balovaptan as a “breakthrough,” to speed research. Preliminary results have been based on a study of male adults with moderate to severe autism. Participants showed some improvement in socialization and communication and tolerated the drug well. Swiss drug company Roche is now beginning to test its effects on children.

“Individuals with autism live with a significant burden due to social impairments and, potentially, other difficulties. These include psychiatric disorders and cognitive deficits, in particular executive dysfunction [such],” says Robert Wisner-Carlson, a psychiatrist with Sheppard Pratt Health System in Baltimore, MD.

“Treatments, including medications, to improve these impairments can possibly change the developmental course [of] and allow for much higher functioning later in life,”  he says. Before recommending balovaptan—if it is ultimately approved— Wisner-Carlson would want to see further studies showing significant improvement in areas like eye contact, social language and interest in socializing.

What’s most interesting about balovaptan to Thomas Frazier, chief science officer for Autism Speaks, is that it’s expected to influence parts of the brain important for social behaviour.

The drug seems to affect vasopressin, a hormone made in the brain that regulates social and emotional function. Vasopressin has been linked specifically to social and sensory processes that are typically impaired in people on the spectrum.

Evdokia Anagnostou, a developmental pediatrician and autism researcher at Holland Bloorview Kids Rehabilitation Hospital in Toronto, will be following the progress of balovaptan research.

“It has shown early promise in randomized, controlled trials [one]. We are waiting for the final, larger of such studies to confirm whether it works as suspected,” she says.


In the meantime, the few medications prescribed to people with autism only treat symptoms of irritability, aggression and anxiety.

“Risperidone and aripiprazole have FDA indication for treating irritability in kids with ASD. However, they have severe side effects, such as significant weight gain, metabolic complications and sedation. And there are no randomized, controlled trials to evaluate drugs to treat anxiety in kids with autism, despite anxiety being one of the most common co-occurring conditions,” says Anagnostou.

She is among those practitioners who’d like to see more research into treating the full range of symptoms that may come with an autism diagnosis and is encouraged by this latest drug development attempt.

Says Frazier: “Particularly for higher-functioning individuals, social difficulties are often a factor in limiting their success in school, an occupation, and everyday interaction.

“Having medications that improve their motivation to engage with others, and, as result, their ability to perceive others, would be a major advance for helping people with autism navigate the social world.”


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